Evidence map›Paper›PMID 41291238›Full record

ReviewNature reviews. Chemistry2025

Chemical strategies for brain delivery of genomic therapy.

Haoyuan Li, Changyue Yu, Tamara Markovic, Eric J Nestler, Yizhou Dong

Abstract readReview
In one paragraph

Review in Nature reviews. Chemistry, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Haoyuan LiDepartment of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Changyue YuDepartment of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Tamara MarkovicFriedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Eric J NestlerFriedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. eric.nestler@mssm.edu.ORCID 0000-0002-7905-2000
Yizhou DongDepartment of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.ORCID 0000-0001-5786-0659

Funding

Transcription Factors in Stimulant and Opioid ActionP01DA047233 · NIDA · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI YASMIN L. HURD · 2019 to 2026
$16.5M
Molecular Studies of Cocaine Action in BrainR01DA007359 · NIDA · YALE UNIVERSITY · PI NESTLER, ERIC J. · 1991 to 2025
$5.6M
Construction of in vivo mRNA delivery systemsR35GM144117 · NIGMS · OHIO STATE UNIVERSITY · PI Yizhou Dong · 2022 to 2026
$2.4M
NIDA NIH HHS P01 DA047233NIDA NIH HHS R01 DA007359NIGMS NIH HHS R35 GM144117
6 · The paper itself

Abstract

Genomic therapy has emerged as a transformative strategy for the prevention, diagnosis and treatment of a wide array of diseases, including Alzheimer's disease, amyotrophic lateral sclerosis and other CNS-related diseases. Recent developments in chemical strategies and delivery platforms have enhanced the potential of genomic therapies for brain disorders. In this Review, we summarize such strategies, focusing on advances in delivery platforms such as lipid nanoparticles, polymers and oligonucleotide conjugates to facilitate the brain delivery of DNA-based or RNA-based therapeutics into the CNS. We present an overview of the chemical structures and functional moieties of lipids, polymers and oligonucleotides used in these platforms. Lastly, we provide an outlook on future chemical directions to further improve the delivery of genomic medicines to the brain.

Indexed as

BrainDrug Delivery SystemsGenetic TherapyGene Transfer TechniquesAnimalsHumansLipidsNanoparticlesOligonucleotidesPolymersLipidsOligonucleotidesPolymers

Identifiers

PMID41291238
PMCPMC12831201

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.